Li-Fraumeni syndrome (LFS) is a genetic disorder characterized by inherited mutations in the p53 tumor suppressor gene. This abnormal p53 function is directly addressed by Advexin treatment.
“In addition to providing targeted therapy for Li-Fraumeni cancer patients who need novel treatments, the results of our study defined important markers to guide Advexin therapy of other cancers with p53 abnormalities that are very common in most types of cancer,” said Dr Neil Senzer, scientific director at the Mary Crowley Medical Research Cancer Center, and the lead clinical investigator of the study.
Treatment of a tumor in an LFS patient with Advexin led to improvement of tumor-related symptoms and resulted in a complete response as determined by positron emission tomography (PET) scans.
The data were presented at the annual meeting of the American Society of Gene Therapy.